对近来有关基因修饰猪作为人类疾病模型的研究进行综述。建立疾病动物模型有利于探索疾病的致病机制,开发治疗药物和诊治途径,是人类疾病临床前研究的重要环节,对实验到临床(B2B)的转化医学研究至关重要。通过基因修饰手段获得的带有人类疾病的模型动物具有遗传稳定性和来源的可重复性,对相应人类疾病的研究更具有举足轻重的作用。由于与人类生理特征具有诸多相似性,猪可以作为良好的人类疾病动物模型,并且比传统的啮齿类疾病动物模型更具有优势。随着大动物胚胎操作技术和基因操作技术的日渐成熟,近年来对通过基因修饰猪建立的人类疾病模型的研究进展也逐步加快。

The animal models of human disease could facilitate the studies of disease pathogenesis and the development of new drugs and treatments. It is an essential step in the preclinical research and the translational medicine studies of Bench to Bedside(B2B). The animal models established by the genetic modification are paid particular attentions due to their genetic stability and repeatable availability. Due to the many similarities between pig and human in physiological characteristics, pig can be a good animal model of human diseases with many advantages over the conventional rodent model. The human disease model using genetically modified pigs sees great progress along with the development of embryo manipulation technologies and genetic modification technologies in large mammals. In this paper, the research advances on genetically modified pigs for human disease models in recent years are reviewed.